Cellectis S.a. · EURONEXT:ALCLS
Cellectis — Investor Presentation (June 2021)
23 slides from Cellectis's investor presentation, Jun 10, 2021
Slide 1: Commitment to a Cure – Investor Presentation June 2021
COMMITMENT TO A CURE Investor Presentation June 2021 cellectis.com
Slide 2: Forward-Looking Statements
FORWARD-LOOKING STATEMENTS This presentation contains “forward-looking” statements These forward-looking statements are made in light of within the meaning of applicable securities laws, including information currently available to us and are subject to the Private Securities Litigation Reform Act o
Slide 3: Writing the History of Allogeneic CAR T-Cells
WRITING THE HISTORY OF ALLOGENEIC CAR T-CELLS 21 years 9 years 8 clinical trials of expertise in of experience in allogeneic ongoing as of 2021; gene editing CAR-T manufacturing 3 Cellectis-sponsored 7 partnered INVENTORS / PIONEERS OF GENE EDITING & ALLOGENEIC CAR T-CELLS In 2012 . . In 2015 . . Mi
Slide 4: Advantages of Allogeneic vs. Autologous CAR-T
ADVANTAGES OF ALLOGENEIC VS. AUTOLOGOUS CAR-T Autologous process: Manufacturing variability + several weeks before treatment is available 1. 2. 3. 4. CANCER TREATMENT CANCER MANUFACTURING OF A INDIVIDUAL CAR-T DECISION PATIENT APHERESIS SINGLE PATIENT THERAPY PRODUCT Allogeneic process: Consistent m
Slide 5: UCARTs – Allogeneic CAR T-Cells Through Precision Gene Editing
UCARTs – ALLOGENEIC CAR T-CELLS THROUGH PRECISION GENE EDITING RATIONAL Engineering EFFECTIVE via accurate insertion Persistence of best CAR via knock-out of CD52 or b2-microglobulin UCART Universal Chimeric Antigen Receptor T-cell TUMOR CELL SMART SAFE Action Administration CAR linked to suicide sw
Slide 6: TALEN: A Versatile Gene Editing Platform
TALEN®: A Versatile Gene Editing Platform X KI with KO Gene KO Gene Repair Gene Gene insertion X replacement Nuclease KI without KO Base editor KI with controlled expression Gene KO Gene Repair
Slide 7: UCART Manufacturing
UCART MANUFACTURING 9 years of experience in allogeneic CAR-T manufacturing Frozen Thawed PBMCs Lentivector TALEN®-mediated leukopaks transduction gene editing 1 2 3 4 Cell expansion Purification Fill & Finish Frozen UCART products 5 6 7 8 P7
Slide 8: Partnerships with Industry Leaders: CAR-T Partnerships
PARTNERSHIPS WITH INDUSTRY LEADERS CAR-T Partnerships Up to $3.2B in potential milestone payments plus royalties Partner License Geography Most Advanced Targets Status Economics to Cellectis Exclusive license to CD19- directed allogeneic CAR T- Ex-US Up To $410M In Ph1 Cells ALLO-501 Development & A
Slide 9: Partnerships with Industry Leaders
PARTNERSHIPS WITH INDUSTRY LEADERS Partnerships- leveraging our TALEN® gene editing platform with additional cell types. Partner License GeographyMost Advanced Targets Status Economics to Cellectis Exclusive license Undisclosed agreement to use Development & specific TALEN® Worldwide Undisclosed Pre
Slide 10: CAR T-Cell Pipeline: Innovative Cancer Therapies
CAR T-CELL PIPELINE: INNOVATIVE CANCER THERAPIES Product Disease Study Phase 1 Phase 1 Pivotal Phase2 Discovery Pre-Clinical Dose Escalation Dose Expansion Partner ACUTE UCART22 LYMPHOBLASTIC BALLI-01 LEUKEMIA ACUTE MYELOID AMELI-01 UCART123 Wholly-controlled LEUKEMIA Product Candidates UCARTCS1 MUL
Slide 11: HSC Genome Surgery Platform
HSC GENOME SURGERY PLATFORM Leukophoresis & Expansion HSC • 75-96% modification High High efficiency in multiple loci Genome Modification Cell Viability Surgery • >80% cell viability & Optimized Efficiency & Potency preserve differentiation Process potential • >4-fold increase in primitive Clinical-
Slide 12: .HEAL Pipeline - Fully Owned by Cellectis
.HEAL PIPELINE - fully-owned by Cellectis HSC genome surgery FOR multiple genetic DISEASES Candidate Indication Cell Type Target Discovery Pre-Clinical IND-enabling TALGlobin-01 Sickle Cell Anemia CD34+ HBB TalX-05 Lysomal Storage Undisclosed Disease CD34+ TalX-03 Primary T-Cell Immunodeficiency STA
Slide 13: UCART22: Initial Anti-Leukemic Activity in BALLI-01
UCART22: Initial Anti-Leukemic Activity in BALLI-01 Phase 1 in R/R B-ALL PHASE 1 dose escalation in N=5 (safety) R/R Adult B-Acute N=5 (efficacy) Lymphoblastic Leukemia BALLI-01 Study Efficacy – Secondary Objective Preliminary data from 5 patients who received UCART22 at DL1 Patients at DL1 achieved
Slide 14: UCART20x22 – Dual-Targeted CAR T-cell for Lymphoma
UCART20x22 – a Dual-Targeted CAR T-cell product candidate for Lymphoma CD20-CAR § Alternative to single target: CD19 CD22-CAR § CD22 and CD20, both validated targets in B cell malignancies § Expected to prevent target escape and strengthen contact TALEN® attributes: TALEN® for TRAC and CD52 KO, same
Slide 15: UCARTMESO – Allogeneic CAR T-Cell in Solid Tumors
UCARTMESO – ALLOGENEIC CAR T-CELL PRODUCT CANDIDATE IN SOLID TUMORS rLV MESOTHELIN IS AN ATTRACTIVE For CAR SOLID TUMOR TARGET First TALEN® KO expression Mesothelin TCR- Graft vs Host CAR ü Mesothelin is a tumor-associated Prevention antigen broadly overexpressed on TCR various malignant tumor cells
Slide 16: Outsmarting the Tumor with Gene Edited CAR T-Cells
OUTSMARTING THE TUMOR WITH GENE EDITED CAR T-CELLS Immunosuppressive cells and factors Physical barriers Intratumoral Negative regulation of milieu effector cells Treg B cell CAF cytokines M2 macrophage Myeloid-derived Extracellular suppressor cell matrix P16 * UCARTMUC1 product candidate is under p
Slide 17: Next-Gen CAR T-Cells – UCARTMUC1 with Multiple Edits
NEXT-GEN CAR T-CELLS – UCARTMUC1* PRODUCT CANDIDATE WITH MULTIPLE EDITS rLV 3 TALEN® Knock-Out TCR KO: MUC1 CAR DNTGFβR2 For CAR Graft vs Host Prevention expression 2 Knock-In TCR TGFBR2 Dominant negative B2M KO: TGFBR2 MHC-I DB2MHLAE Resistance to patient’s DPD1-IL12 T-cells HLA-E knock-in PD1 Resi
Slide 18: Overcoming 'Cold Tumor' Obstacle by Targeting CAFs
OVERCOMING ‘COLD TUMOR’ OBSTACLE BY TARGETING CAFS § ECM deposition forming physical barrier § Cytokine secretion forming chemical barrier Cancer-associated Fibroblasts (CAFs): central role for T-cell exclusion and immune suppression in solid tumors P18
Slide 19: UCARTFAP: Allogeneic CAR-T for FAP+ Solid Tumors
UCARTFAP: AN ALLOGENEIC CART PRODUCT CANDIDATE FOR FAP+ SOLID TUMORS rLV control UCARTFAP For CAR expression TCR KO: Graft vs Host FAP CAR Prevention FAP-CAR 2.11% 74.8% TCR FSC-H FAP-CAR MHC-I B2M KO: Resistance to TCRa/b patient’s T cells 58.9% MHCI P19 * UCARTFAP product candidate is under early
Slide 20: A Cross-Atlantic Organization
A CROSS-ATLANTIC ORGANIZATION US Footprint EU Footprint New York, NY - Gene Editing Platform - Innovation - Clinical Development Paris, France - Process & Analytical Paris Development - Starting Materials Manufacturing • 14,000 sq ft. facility • Manufacturing operations went live in 2020 Raleigh, No
Slide 21: The Cellectis Group
THE CELLECTIS GROUP ~64.5%* ownership NASDAQ: CLLS | EURONEXT GROWTH: ALCLS NASDAQ: CLXT • ~$211M** cash and cash equivalents, not including $45.5M • $20M* cash and cash proceeds through ATM program in April 2021 equivalents • Cash runway into early 2023 • Cash runway into H2 2022 • Patient focused
Slide 22: Achieved Milestones and Current Clinical Status
ACHIEVED MILESTONES Proprietary clinical programs Partnered clinical programs Manufacturing UCART22: Phase 1 in R/R ALL ongoing; ALLO- 501/ ALLO- 501A1: Phase 1 in R/R NHL 2 in-house GMP manufacturing plants: data presented at ASH 2020 ongoing; data will be presented at ASCO 2021 Paris, France facil
Slide 23: Thank You
THANK YOU Reach us at: investor@cellectis.com Cellectis Paris Cellectis New York Cellectis Raleigh 8, rue de la Croix Jarry 430 East 29th Street 2500 Sumner Boulevard 75013 Paris – France 10016 New York, NY – USA 27616 Raleigh, NC – USA P23